Sangamo Therapeutics Aligns with FDA on Accelerated Approval Pathway for ST-920 Gene Therapy in Fabry Disease

Alignment with FDA:
Sangamo Therapeutics has announced an alignment with the FDA on an accelerated approval pathway for ST-920, a gene therapy candidate for Fabry disease, with a Biologics License Application (BLA) submission expected in the second half of 2025.

Accelerated Approval Basis:
The FDA has agreed that the estimated glomerular filtration rate (eGFR) slope data at one year across all Phase 1/2 patients can serve as the primary basis for approval under the Accelerated Approval Program.

No Additional Registrational Study:
This approach eliminates the need for an additional registrational study, potentially expediting the approval timeline by approximately three years.

Data Availability:
The complete dataset to support the Accelerated Approval pathway will be available in the first half of 2025.

Clinical Significance:
Fabry disease is a debilitating condition with significant unmet medical needs, and the potential approval of ST-920 could address many aspects of the disease.

Ongoing Activities:
Sangamo is preparing for BLA readiness activities for isaralgagene civaparvovec while continuing business development discussions with potential collaboration partners.